Gene Therapy Trial for CLN6 Batten Disease

Part of paid clinical trials in La Jolla, California.

Sponsor
The Charlotte and Gwenyth Gray Foundation
Study ID
NCT07582484
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
4 Months - N/A
Healthy Volunteers
Not accepted

Interventions

  • scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) — DRUG
    self-complementary adeno-associated viral vector, serotype 9 (scAAV9), which contains the human CLN6 gene under the control of a hybrid CMV/CB promoter

Study Details

The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms. Participants will: Receive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study

Key Dates

Start date
Aug 31, 2026
Status verified
May 2026
Primary completion
Aug 31, 2027
Completion
Aug 31, 2028

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment group

Primary Outcome Measure

Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0 [ Time Frame: From informed consent through Month 24 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of California, San Diego - Rady Children'sLa JollaCalifornia92093
Olivia Kim-McManus, MD
[email protected]
858-576-1700

Find similar trials in La Jolla, CA

By research site
University of California, San Diego - Rady Children's· La Jolla, CA

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